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zzaatt
Friday, 09/14/18 01:10:44 PM
Re: Auto1 post# 15508
0
Post # of 15517
Quote:
Just sound out the big words and then google them
LOL! Putting "big words" in a post does not necessarily add value or coherence, ONLY if they're condign!
Wow.. you are good with the Thesaurus. I can just see you sitting there in your Mom's basement with your Thesaurus and your Netter's dogearing pages as you go.
Quote:
Ariad Alum
Don't you wish you were?????
No, Not as a pharma rep like you. I can only imagine the tens of thousands you must have made... Hows unemployment treating you?
zzatt,
Why were you not retained by Takeda? In M/A's the acquirer typically retains the best and brightest from the acquired...
zzatt...
shouldn't you be out making sales calls instead of SO on your computer? Or, are you on your lunch break?
As stated earlier... These recent data on the endpoints described are WEAK. Please re-read the article and quotes from the principal investigator, and look at the p-values. While 'significant' they are NOT significant enough to be considered 'blockbuster', by a looong shot.
The best evidence that what I'm trying to educate you on are the following:
1) The analysts and investors response was a yawn. Look it up.
2) The PI highlighted 'walking distance / delay to amputations' - Do you really think these are the endpoints that PSTI hopes to crush?!?! Looks to me like grasping for straws.
3) 'Changes' to the PIII study, not an expedited conclusion. Looks like PSTI is getting a 101 on trial design.
Inexperience is far more expensive to hire than Highly experienced...
zzaatt
Friday, 09/14/18 09:57:14 AM
Re: Auto1 post# 15498
0
Post # of 15508
Quote:
Actually, these endpoints are weak from a reimbursement standpoint.
Actually, the only "endpoint" that is significant for approval is the one defined as success by the FDA/EMA/etc. Reimbursement is a policy issue and it comes much later.
Quote:
The type of results that it will take to get from fast track to expedited review need to look like 1) Reductions in amputations - patients requiring no longer requiring 2) Patients coming off of disability and returning to work 3) Revascularization via angiogenesis, verifiable through imaging, etc.
1) BINGO, we got it, reductions in amputations
2) FDA does not examine changes in patient's life style or change in work habits. Many of the relevant patients are elderly and retired. We're looking for relief, from pain, amputation, death, massive cost of care. That's precisely what PLX-PAD does.
3) BINGO, we got that too.
Looks like Pluristem is off and running! We're getting closer to the target by the day!
Could you rephrase that, I can't extract any coherent point! My best guess is that it's something to do with the P3 for CLI.
Just sound out the big words and then google them, Ariad Alum.
"My fellow Ariad alumni" - THIS explains everything.
And think about it... Truth be told,
This indication, at the time of trial design was PSTI's highest opportunity of success. Higher than any other possible indications based on the MOA (mechanism of action) - mesenchymal stem cell activity, healing and regenerating - the 'promise' of stem cell treatment. CLI has the Highest chance of success for this MOA to demonstrate its most statistically significant effects. Literally, angiogenesis and vasculogenesis … growing new tissue, vessels, capillaries, in essence, re-birthing tissues, vasculature and limbs.
Now, re-read the primary investigators quotes, do you get the sense that this type of success is taking place in the trial????
And now... Study Changes and reaffirmation of 2020 as end of study. Think about it, read it for yourself.
Actually, these endpoints are weak from a reimbursement standpoint. Unfortunately insurance companies could care less about improvement ins number of steps. 83% improvement means what? What real benefit to the system? I get it, its very meaningful for the patient that can walk further, but are they back to work? Off gov't assistance? Disability? Likely not. Furthermore, the delay to amputations and revascularization are likely not statistically significant enough, although statistically significant.
The type of results that it will take to get from fast track to expedited review need to look like 1) Reductions in amputations - patients requiring no longer requiring 2) Patients coming off of disability and returning to work 3) Revascularization via angiogenesis, verifiable through imaging, etc. Without these, commanding a price point for 'stem cell' type activity will be virtually impossible. P-values in the .005 range IMHO
Thanks for making my point with your non-answer answer. IT was, in fact, a real question for you, with a question mark and all.
zzatt, still waiting on your response...
You can't have it both ways. You reject my explanation for 'NO CONTRACT', yet you claim that all requirements have been met to have a contract...
Not 1 single military hospital: Bethesda, Walter Reid, Quantico, etc., etc.
Again, what say you? Why do we NOT have a contract for R-18, for as you say, a perfectly good delivery device, 'vial & syringe' right now?
Outstanding summation, Mikems.
The good news is that Zami & Yaky are now being held accountable. NOT DOUBT, Dr. Jeffs was instrumental in re-negotiating their 'consultants agreements'
Easy and inexpensive termination clauses! Well done BOD!
Still think 1 CEO and 1 CCO, both with REAL commercialization experience would be the prudent investment vs. current arrangement. IMHO.
I'm sure the ATM share purchasers were more than happy to be 'shorted' by PSTI... Great move to build investors.
As of June 30, 2018, we had sold 3,599,408 shares of common stock at an average price of $1.43 per share.
Let me know when PSTI starts buying its stock back in the open market. That would actually signal confidence...
Incomparable... Apples and Oranges when it comes to death and endpoints studied.
There you have it folks!
Zzaatt cannot produce an original thought, only criticize factual posts. All Hat, no cattle.
One of the things I like most about Spidey is he never talks.
Just get home from school?
Let me guess... You are leaving that decision to management...
Waiting...
Yes. What is your answer to the question?
Oh yeah, I forgot, can't produce enough...
You can't have it both ways.
Funny, I have been saying the current delivery system presented a logistical 'in-theatre' issue for over a year now... and was likely the reason there is NO contract with the DOD.
This confirms it, and will also now extend the timeline to approval, as these 'super syringes' will have to be used in a trial to prove = to ship & thaw vials...
I'm glad you agree with me now allo. You debated the point vigorously in the past. Pass the waffles.
And population control is that of the Gov't. US Gov't can't afford Medi/Medi with a life expectancy of 75+/-. WHY would they support living longer?
https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm
Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.
Determining whether a condition is serious is a matter of judgment, but generally is based on whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one. AIDS, Alzheimer’s, heart failure and cancer are obvious examples of serious conditions. However, diseases such as epilepsy, depression and diabetes are also considered to be serious conditions.
Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially better than available therapy.
Any drug being developed to treat or prevent a condition with no current therapy obviously is directed at an unmet need. If there are available therapies, a fast track drug must show some advantage over available therapy, such as:
Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
Avoiding serious side effects of an available therapy
Improving the diagnosis of a serious condition where early diagnosis results in an improved outcome
Decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment
Ability to address emerging or anticipated public health need
A drug that receives Fast Track designation is eligible for some or all of the following:
More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA
Fast Track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and make a decision within sixty days based on whether the drug fills an unmet medical need in a serious condition.
Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
Should be approaching the deadline for the FDA's decision then. Generally must be reviewed and made within 15 months!
Lets pray so, they are not treating anyone who could benefit VIA the EAP it would appear.
How Sad.
So... IS it Patient SAFETY or PROFITS that 'Management' is basing these decisions on?
Actually, I Am. More qualified than Mr. Yaki Yanay. THAT I can tell you.
IF you read my posts on the process and due-diligence that is required for compassionate use, you would see that it is under 'informed-consent' and the their is as much OR MORE known about the patient who consents than some trial patients.
Its the Martin Skreli's of the world who choose profits over patients that eventually meet their demise.
But then again, maybe the products are not as safe or effective as touted...Yaki would know that! IMHO.
zzaatt
Monday, 09/10/18 03:07:44 PM
Re: Auto1 post# 15404
zzatt, you never learn, you don't even consider what you are posting either. You just made the assertion that it is 'RISKY', but you also shared an experience of 'DEATH' in a 'clinical trial', I'm assuming it was one you approved of and "management decided on" - but, they still died... This is how clinical trials go... You can't control it, you just double spoke by supporting kiki's approach to a purified, trial-only protocol.
PS - the death, if ruled unrelated, doesn't count against the trial data. companies are prepared (if their leadership is good) for these unfortunate situations. Give us ALL a break and lay off the Vyvanse.
Post # of 15414
Quote:
Right kiki27...
So the FDA doesn't know what it is doing to authorize an early access program. Shame on them, they should know better.....
kiki was correct, and your sarcasm contains no useful information!
My response to kiki's original post:
Absolutely right! Dangerous for patients, and for the success of the trial. I have seen p3 clinical trials shut down when a very sick patient died, the program was shut down for months, then we find out that the cause of death had nothing to do with the drug in question. For a small biotech like Pluristem that could be devastating. It would also delay approval, and thus access for patients who would actually benefit.
Millions... Please tell me this board is more educated than this... How EVERYONE is not on the BANDWAGON demanding answers on this is mind numbing... Could it be Poor Leadership?
https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM351264.pdf
Charging for Investigational Drugs Under an IND —
Questions and Answers
Guidance for Industry
[color=red][/color]
U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER)
June 2016 Procedural
Charging for Investigational Drugs Under an IND — Questions and Answers
Guidance for Industry
[color=red][/color] Additional copies are available from:
Office of Communications, Division of Drug Information Center for Drug Evaluation and Research Food and Drug Administration 10001 New Hampshire Ave., Hillandale Bldg., 4th Floor Silver Spring, MD 20993-0002 Phone: 855-543-3784 or 301-796-3400; Fax: 301-431-6353 Email: druginfo@fda.hhs.gov http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm
and/or
Office of Communication, Outreach and Development Center for Biologics Evaluation and Research Food and Drug Administration 10903 New Hampshire Ave., Bldg. 71, Room 3128 Silver Spring, MD 20993-0002 Phone: 800-835-4709 or 240-402-8010 Email: ocod@fda.hhs.gov http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm
U.S. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER)
June 2016 Procedural
Contains Nonbinding Recommendations
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TABLE OF CONTENTS
I. INTRODUCTION............................................................................................................. 1 II. BACKGROUND ............................................................................................................... 2 III. QUESTIONS AND ANSWERS ....................................................................................... 2 A. General Questions .......................................................................................................................... 2 B. Charging in Clinical Trials ........................................................................................................... 3 C. Charging For Expanded Access Use ............................................................................................ 6 D. Cost Recovery Calculations .......................................................................................................... 7
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Charging for Investigational Drugs Under an IND — Questions and Answers Guidance for Industry1
This guidance represents the current thinking of the Food and Drug Administration (FDA or Agency) on this topic. It does not establish any rights for any person and is not binding on FDA or the public. You can use an alternative approach if it satisfies the requirements of the applicable statutes and regulations. To discuss an alternative approach, contact the FDA staff responsible for this guidance as listed on the title page.
I. INTRODUCTION
This guidance provides information for industry, researchers, physicians, institutional review boards (IRBs), and patients about the implementation of FDA’s regulation on charging for investigational drugs2 under an investigational new drug application (IND) for the purpose of either clinical trials or expanded access for treatment use (21 CFR 312.8), which went into effect on October 13, 2009.3 Since 2009, FDA has received a number of questions concerning its implementation of the charging regulation. As a result, FDA is providing guidance in a question and answer format, addressing the most frequently asked questions. In a separate guidance,4 FDA provides answers to questions concerning regulations on expanded access to investigational drugs for treatment use (21 CFR part 312, subpart I), which also went into effect on October 13, 2009. Also in a separate guidance, FDA describes Form FDA 3926 (Individual Patient Expanded Access--Investigational New Drug Application (IND)) and the process for submitting expanded access requests for individual patient INDs.5
1 This guidance has been prepared by the Office of Medical Policy in the Center for Drug Evaluation and Research in cooperation with the Center for Biologics Evaluation and Research at the Food and Drug Administration.
2 For the purposes of this guidance, the terms investigational new drug, investigational drug, drug, and drug product refer to both human drugs and biological products regulated by the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research.
3 Federal Register of August 13, 2009 (74 FR 40872).
4 See the guidance for industry Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers for the Agency’s current thinking on this topic. We update guidance documents periodically. To make sure you have the most recent version of a guidance, check the FDA guidance Web page at http://www.fda.gov/RegulatoryInformation/Guidances/.
5 See the guidance for industry Individual Patient Expanded Access Applications: Form FDA 3926 for the Agency’s current thinking on this topic.
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In general, FDA’s guidance documents do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required.
II. BACKGROUND
For many years, FDA authorized charging for an investigational drug under a regulation that was published in 1987 (the 1987 charging rule) (52 FR 19466, May 22, 1987). In 2009, FDA revised its 1987 charging rule for three principal reasons: (1) to take into account circumstances concerning charging for investigational drugs in a clinical trial that were not anticipated when the rule was written, (2) to set forth criteria for charging for investigational drugs made available under all categories of expanded access described in the expanded access regulations that were also revised in 2009, and (3) to specify the types of costs that can be recovered when charging for an investigational drug under an IND.
The revised charging regulation provides the following:
• General criteria for authorizing charging for an investigational drug (21 CFR 312.8(a)) • Criteria for charging for an investigational drug in a clinical trial (21 CFR 312.8(b)) • Criteria for charging for an investigational drug for an expanded access use under 21 CFR part 312, subpart I (21 CFR 312.8(c)) • Criteria for determining what costs can be recovered when charging for an investigational drug (21 CFR 312.8(d))
III. QUESTIONS AND ANSWERS
A. General Questions
Q1: How much time does FDA have to review and respond to a request to charge for an investigational drug?
A1: The provision in 21 CFR 312.8 does not specify a time frame for FDA to respond to a request to charge for an investigational drug. However, FDA intends to respond to charging requests within 30 days of receipt when possible.
Q2: Under 21 CFR 312.8, who requests authorization from FDA to charge for an investigational drug for use under an IND?
A2: Section 312.8 permits only the sponsor of the IND to request FDA’s authorization to charge for an investigational drug for use under the IND (§ 312.8(a)). The sponsor of the IND is not always the manufacturer of the drug. For example, if the manufacturer of an unapproved drug is
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not the sponsor of the IND under which the drug will be used, the manufacturer is not required to obtain authorization from FDA to charge the sponsor of the IND for the unapproved drug. However, in such a situation, if the sponsor wants to charge patients to recover the cost charged by the manufacturer, the sponsor must obtain FDA’s written authorization before it can begin charging patients (§ 312.8(a)(3)).
Q3: Once FDA authorizes a request to charge, whom may the sponsor charge?
A3: Although FDA determines whether a sponsor may charge for an investigational drug used in a clinical trial or for expanded access, FDA does not decide how that charging is to be carried out. FDA anticipates that the sponsor would ordinarily charge a patient directly or would charge a third-party payer if reimbursement were available. FDA notes that it has no authority to require that the Centers for Medicare and Medicaid Services reimburse for investigational drugs for which FDA has authorized charging. Similarly, FDA has no authority to dictate reimbursement policy to any other entity, including private health insurance providers. For questions pertaining to third-party payer reimbursement, the third-party payer should be consulted.
B. Charging in Clinical Trials
Q4. When a sponsor uses its own investigational drug in a clinical trial, what requirements must the sponsor satisfy to charge for the drug?
A4: When a sponsor is using its own investigational drug, including an investigational use of its approved drug, in a clinical trial, a sponsor must do all of the following to obtain authorization to charge for the drug:
• Provide evidence to FDA that the drug has a potential clinical benefit that, if demonstrated in clinical investigations, would provide a significant advantage over available products in the diagnosis, treatment, mitigation, or prevention of a disease or condition (21 CFR 312.8(b)(1)(i)). • Demonstrate that the data to be obtained from the clinical trial would be essential to establishing that the drug is effective or safe for the purpose of obtaining initial approval, or would support a significant change in the labeling of an approved drug (e.g., a new indication, inclusion of comparative safety information) (§ 312.8(b)(1)(ii)). • Demonstrate that the clinical trial could not be conducted without charging because the cost of the drug is extraordinary to the sponsor (§ 312.8(b)(1)(iii) (see also Q5 regarding extraordinary cost). • Provide documentation to support its calculation for cost recovery to show that the calculation is consistent with the requirements of § 312.8(d)(1). The documentation must be accompanied by a statement that an independent certified public accountant has reviewed and approved the calculation (§ 312.8(d)(3)).
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Sponsors must meet all of these requirements and must obtain written authorization from FDA to charge before they begin to charge for an investigational drug (§ 312.8(a)(3)).
Q5: What constitutes extraordinary cost?
A5: As noted in A4, 21 CFR 312.8(b)(1)(iii) requires that the sponsor demonstrate that it could not conduct the clinical trial without charging for the investigational drug because the cost of the drug is extraordinary to the sponsor. Section 312.8(b)(1)(iii) also describes the reasons that the cost of a drug may be extraordinary. The cost of a drug may be considered extraordinary to a sponsor because of manufacturing complexity, scarcity of a natural resource, the large quantity of the drug needed (e.g., based on the size or duration of the trial), or some combination of these or other extraordinary circumstances (e.g., resources available to a sponsor) (§ 312.8(b)(1)(iii)).
Q6: Does FDA consider the financial resources available to a sponsor when determining whether the cost of providing its investigational drug in a clinical trial is extraordinary?
A6: Yes. The provision in 21 CFR 312.8(b)(1)(iii) describes the reasons that the cost of a drug might be extraordinary to the sponsor, including the resources available to a sponsor. For example, a cost that is considered extraordinary to a small start-up company may not be considered extraordinary to a large established company.
Q7: What is an independent certified public accountant?
A7: An independent certified public accountant is a certified public accountant who is not an employee of the company seeking to charge for an investigational drug.
Q8: When a company is the sponsor of a clinical trial evaluating an unapproved use of its approved drug, is the company required to obtain authorization to charge for its drug?
A8: Yes. In accordance with 21 CFR 312.8(b)(1), a sponsor of a clinical trial must obtain authorization to charge for its own drug, including investigational uses of its approved drug.
Q9: If a sponsor (e.g., a physician-researcher who is a sponsor-investigator) purchases an approved drug from the company that markets the drug or another commercial distribution entity (e.g., a pharmacy or a wholesaler) for use in a clinical trial, is the sponsor required to obtain authorization from FDA to charge for the approved drug?
A9: No. If a sponsor is not the company that markets the approved drug and the sponsor must purchase the approved drug for use as part of the clinical trial evaluation (e.g., in a clinical trial of a new use of the approved drug or for use of the approved drug as an active control) or as concomitant therapy, the sponsor is not required to obtain FDA authorization to charge for the approved drug (see 21 CFR 312.8(a)(1)).
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Q10: If a sponsor’s own approved drug is used as concomitant therapy for an approved use during a clinical trial intended to evaluate another drug, should the sponsor obtain authorization to charge for the drug used as concomitant therapy?
A10: No. In many clinical trials, approved drugs are used as concomitant therapy for subjects during the trials, but are not part of the clinical trial evaluation. For example, (1) patients may be required by a protocol to take certain approved drugs as concomitant therapy before or during the trial (e.g., patients may receive antihistamines for immune response concerns in a clinical trial to study a recombinant protein, in order to mitigate potential risks of participation in the trial; or all patients may receive concomitant therapy before randomization to either the investigational drug or placebo) or (2) patients may be permitted by the protocol to continue taking certain approved drugs as concomitant therapy during the trial because such drugs are not likely to interact with the study drug(s) or otherwise confound the results of the trial (e.g., pain medications for patients in a clinical trial to study a drug intended to treat cancer) or because discontinuing the drug might adversely affect the patient.
In accordance with 21 CFR 312.8(b)(1), a sponsor must obtain prior authorization from FDA to charge for its investigational drugs, including investigational uses of its approved drugs. However, FDA regulations do not require a sponsor to obtain prior authorization to charge for its own approved drug when that drug is used as concomitant therapy for an approved use and is not part of the clinical trial evaluation.
Q11: How can a sponsor charge for its investigational drug in a blinded, controlled clinical trial without compromising the blind and therefore the integrity of the clinical data generated from the trial?
A11: FDA recognizes that in certain situations, charging for an investigational drug in a clinical trial may have the potential to compromise the blinding of study subjects to which therapy they have received (e.g., in a situation in which subjects who are in the treatment arm of the study are charged, and subjects who are in the control arm are not charged). However, FDA believes that there are methods for preserving the blind that sponsors could use in most cases. We are not providing hypothetical examples because we anticipate that the method for preserving the blind will be unique to each study design. When these situations arise, the sponsor may seek advice from the appropriate review division in the Office of New Drugs (OND) in the Center for Drug Evaluation and Research (CDER) or from the review office in the Center for Biologics Evaluation and Research (CBER) on how to preserve the blind, based on the specifics of the given situation. To find the appropriate CDER OND review division, see http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm42 9610.htm. For contact information for CBER, see http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/uc m106001.htm.
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Q12: How long may a sponsor charge for an investigational drug in a clinical trial after FDA authorizes the charging?
A12: Charging may continue for the entire length of the clinical trial unless FDA specifies a shorter duration (21 CFR 312.8(b)(2)).
C. Charging For Expanded Access Use
Q13: What requirements must a sponsor satisfy to charge for expanded access use?6
A13: The sponsor of an expanded access IND or protocol must do all of the following to obtain authorization to charge for the drug:
• Provide reasonable assurance to FDA that charging will not interfere with drug development (21 CFR 312.8(c)(1)). • Provide documentation in its charging request submission to show that its calculation of the amount to be charged is consistent with the requirements in § 312.8(d). This documentation must be accompanied by a statement that an independent certified public accountant has reviewed and approved the calculation (§ 312.8(d)(3)). Documentation of the calculation of the amount to be charged for a drug obtained from another source could consist of a copy of the receipt or invoice from the source that provided the drug to the expanded access sponsor. For expanded access under § 312.320 (treatment IND or treatment protocol), the reasonable assurance that charging will not interfere with drug development must include (1) evidence of sufficient enrollment in any ongoing clinical trials needed for marketing approval to reasonably assure FDA that the trial(s) will be successfully completed as planned; (2) evidence of adequate progress in the development of the drug for marketing approval; and (3) information submitted under the general investigational plan specifying the drug development milestones the sponsor plans to meet in the next year (§ 312.8(c)(2)).
Sponsors of expanded access INDs and protocols must meet these requirements and obtain written authorization from FDA before they begin to charge for an investigational drug (§ 312.8(a)(3)).
Q14: How long may a sponsor charge for an investigational drug for expanded access use after FDA authorizes the charging?
A14: Charging for an investigational drug for expanded access use may continue for 1 year from the time of FDA authorization unless FDA specifies a shorter period (21 CFR 312.8(c)(4)). FDA periodically reassesses whether charging is interfering with development of a drug for marketing 6 The regulations regarding expanded access to investigational drugs for treatment use are in 21 CFR part 312, subpart I. As explained in footnote 4, FDA’s guidance for industry Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers provides information on expanded access.
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and believes that the 1-year anniversary is typically a reasonable point in time to reevaluate charging requests. FDA may reauthorize charging for an investigational drug for expanded access use for additional periods. If a sponsor wishes to continue charging beyond the expiration of the existing authorization, FDA recommends that the sponsor submit a request to reauthorize charging at least 60 days prior to the expiration of the existing authorization to charge for the investigational drug.
Q15: What must a sponsor do to obtain authorization to continue charging for an investigational drug for expanded access use beyond the duration of its existing charging authorization (i.e., for additional periods)?
A15: If a sponsor wishes to continue charging beyond the duration of its existing charging authorization, the sponsor must submit a request to FDA for reauthorization to charge for the investigational drug (21 CFR 312.8(c)(4)). The request must satisfy the same requirements as the initial request for charging authorization (see Q13). It is also helpful for sponsors to specify whether any information from the original or previous request has changed. The sponsor must receive written reauthorization from FDA before it can continue to charge for the investigational drug beyond the period previously authorized (21 CFR 312.8(a)(3)).
D. Cost Recovery Calculations
Q16: What costs can a sponsor recover when charging for an investigational drug in a clinical trial?
A16: A sponsor can only recover the direct costs of making a drug available to subjects in a clinical trial — that is, those costs that are specifically and exclusively attributable to providing the drug to clinical trial subjects (21 CFR 312.8(d)(1)). These include costs to manufacture the drug in the quantity needed to conduct the clinical trial for which charging has been authorized or costs to acquire the drug from another source, including costs to ship and handle (e.g., store) the drug.
Q17: What costs can a sponsor recover when charging for an investigational drug for expanded access use under 21 CFR part 312, subpart I?
A17: When charging for individual patient expanded access (under § 312.310) to an investigational drug, a sponsor may recover only its direct costs associated with making the drug available to the patient (see Q16 and § 312.8(d)). For individual patient expanded access, the sponsor may not charge for indirect costs, including administrative costs associated with providing an investigational drug. Examples of indirect costs include:
• Costs associated with developing the treatment protocol and informed consent document • Costs associated with corresponding with the IRB, FDA, and/or the drug manufacturer • Costs associated with reporting to the IRB and/or FDA • IRB fees and expenses
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When charging for an investigational drug used in an intermediate-size patient population expanded access IND or protocol (under § 312.315) or a treatment IND or protocol (under § 312.320), in addition to the direct drug costs, a sponsor may recover the cost of (1) monitoring the expanded access IND or protocol, (2) complying with IND reporting requirements, and (3) other administrative costs directly associated with the expanded access use (§ 312.8(d)(2)).
Q18. May the sponsor of an expanded access IND or protocol recover the cost of the fees the sponsor pays to a third party for administering an intermediate-size patient population expanded access IND or protocol or a treatment IND or protocol?
A18: Yes. FDA interprets 21 CFR 312.8(d)(2) as permitting the sponsor of an expanded access IND or protocol to recover the cost of the fees paid to a third party for administering an intermediate-size patient population or treatment IND or protocol, including any profit for the third party that may be included in the fees. The fees paid to the third party should be included in the calculation for cost recovery that the sponsor provides in its request to charge.
Q19. Does a sponsor need FDA authorization to charge for the costs of drug delivery, including the costs associated with formulation, packaging, instrumentation, monitoring, disposables, setup, and nursing care?
A19: No. The provision in 21 CFR 312.8(d)(1) is intended to permit a sponsor to recover the direct costs incurred in making a drug available from the onset of manufacturing to the point it arrives at the destination to which it was shipped or, for a drug acquired from another source as a finished product (e.g., when manufacturing is outsourced), acquisition, shipping, and handling costs for the drug. Recovery of subsequent costs incurred at a clinical trial site (e.g., a hospital or clinic), including pharmacy costs (e.g., the cost to reconstitute a drug for infusion), nursing costs (e.g., costs associated with administering a drug and monitoring study subjects), equipment costs (e.g., intravenous administration sets, infusion pumps), and costs for study-related procedures (e.g., chemistry labs, radiographic procedures), do not fall within the scope of § 312.8. In other words, FDA authorization is not needed for a sponsor to recover those costs.
Q20: What information is a sponsor required to submit to support its cost calculation?
A20: Under 21 CFR 312.8(d)(3), to support its calculation of recoverable costs, a sponsor must provide documentation to show that its calculation is consistent with the requirements of § 312.8(d)(1), describing recovery of direct costs and, if applicable, the requirements of § 312.8(d)(2), describing certain additional costs that may be recovered for intermediate-size patient population expanded access uses or treatment INDs or protocols. This documentation must be accompanied by a statement that an independent, certified public accountant has
Again...Reasonable and Pertinent Questions for PSTI. If they are NOT
addressed in the quarterly report specifically and directly, it would be a dereliction of duty by leadership. Period, end of discussion.
Auto1
Tuesday, 09/04/18 03:24:14 PM
Re: None
0
Post # of 15410
Here are 2 very reasonable and pertinent questions that should be answered for all stock-holders ending this quarter:
1.) How many PLX-PAD patients have been treated under the FDA's Expanded Access Program YTD.?
2.) How much revenue has been generated via this program? Private pay, Insurance?
You cannot convince anyone that there are NO wealthy PAD patients that would be knocking the door down at PSTI to try this 'miracle'...
Show us the money!
FACTs: 1.The Drug Company MUST INNITIATE the process.
2. It is ultimately the decision of the Drug Company to supply drug.
Yaki is SO stupid, he throws out revenue/FDA when the company gets the greatest vote of confidence and opportunity it has EVER seen... Real Patient Advocate this average accountant is......
What is the FDA’s role in compassionate use?
[color=red]“Regarding Compassionate Use, it's a common misperception in the medical community that the FDA decides who gets compassionate use exceptions in totality. That is only part true. The first thing that needs to occur is the drug company has to APPLY [make application to the FDA].” Greg Merfeld, 2010
[/color]The FDA cannot compel a company to supply an individual patient with an investigational drug outside of its planned clinical trials. The manufacturer or sponsor makes the final decision to provide an experimental drug or therapy to a patient. The sponsor may consider many factors, including the amount of information available about the drug, the amount of drug available, and how best to use its resources to optimize development of the drug for marketing. This maximizes the availability of the drug to patients who can benefit from it. In some cases, the sponsor is unwilling to provide the product outside of clinical trials, especially relatively early in drug development. Patients are sometimes confused or angered by this situation and misinterpret the company's unwillingness to provide the product as an FDA action.
The FDA may decide not to allow compassionate use treatment because of safety concerns. Generally, however, if a physician makes a request for treatment of an experimental drug with a patient for whom no effective therapy exists while there is an ongoing study of the drug and a sponsor agrees to provide the product, FDA does not typically object to compassionate use treatment.
According to Dr. Temple, “The FDA believes that it is appropriate to make certain promising, but not-yet-approved, products available to patients with serious and life-threatening illnesses who lack alternative treatment. This should be done in a way that does not interfere with recruitment to the clinical trials needed to support the effectiveness and safety of the drug. It should also be done fairly.”
https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm
U.S. Department of Health and Human Services
U.S. Food and Drug Administration
Expanded access (sometimes called “compassionate use”) is the use of investigational drugs, biologics or medical devices outside the clinical trial setting for treatment purposes. Expanded access may be appropriate when all the following apply:
Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
Patient enrollment in a clinical trial is not possible.
Potential patient benefit justifies the potential risks of treatment.
Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication
Information for Patients, Physicians and Industry Patients
Learn about what your physician should do before submitting a request for individual patient expanded access use of an investigational medical product, who may be eligible for expanded access, associated costs, FDA contacts and more[color=red][/color]
Physicians
Learn about your responsibilities under the expanded access pathway, how to submit a request for expanded access for an individual patient (including for emergency use), which forms to use, FDA contacts and more.
Industry
Learn about current regulations, what information is required when you provide access to investigational medical products under an individual patient expanded access IND, and view an example of wording that could be used for a Letter of Authorization, FDA contacts and more.
Key Contact Information
1. During Normal Business Hours (8 a.m. - 4:30 p.m. ET, weekdays)
For specific questions during normal business hours:
Investigational drugs: 301-796-3400 or druginfo@fda.hhs.gov [CDER's Division of Drug Information], or contact the appropriate review division, if known
Investigational medical devices: 301-796-7100 or DICE@fda.hhs.gov [CDRH's Division of Industry and Consumer Education]
Investigational biologics: 240-402-8020 or 800-835-4709 or industry.biologics@fda.hhs.gov [CBER's Office of Communication, Outreach and Development]
For general questions, or if you are unsure of who to contact, contact the Patient Affairs Staff at 301-796-8460 or patientaffairs@fda.hhs.gov.
2. After 4:30 p.m. ET weekdays and all day on weekends
For emergency requests for all medical products (drugs, biologics, and medical devices) contact FDA's Emergency Call Center at 866-300-4374.
For additional contact information and mailing addresses for forms: Expanded Access Contact Information
“This is a true vote of confidence by the FDA in our cell therapy and a landmark achievement for Pluristem and its shareholders. It gives us the ability to begin treatments using our cell product, offering treatment to certain CLI patients who have poor therapeutic options, while also collecting real-world data alongside our ongoing Phase III clinical study,” said Yaky Yanay, Co-CEO and President of Pluristem. “We are hopeful that the FDA may also allow us to be compensated for the costs of treatment, which can support our work developing effective cell therapies for millions of patients worldwide.”
http://www.pluristem.com/wp-content/uploads/2018/01/Expanded_Access_Program_CLI_final_isa.pdf
Right kiki27...
So the FDA doesn't know what it is doing to authorize an early access program. Shame on them, they should know better.....
See what I mean?!
Reasonable discussion buried in babbling nonsense and donkey talk. So predictible...
I wonder what Gary thinks
About a company that has an early access nod from the FDA that is NOT pursuing anyone that could even minimally benefit from a CURE that is 100% safe as has been touted by leadership etal. here on ihub...
PS, the early access nod comes After the FDA determines that the likelihood of 'no harm' exceeds the risk....
We have The CURE, wouldn't we want to help anyone that could benefit? We are 100% safe, right? Zzaatt?
Are you suggesting profits over patients, zzaatt?
The publicity and goodwill is enough isn't it?
Please don't feed the Animals.
Releasing on a holiday is the preferred approach when announcing failures. When nobody is watching.
Maybe allo. Knows something on this one.... Burp.
Further too....
If we are NOT worried about reimbursement, PATIENTS FIRST, reimbursement second..... There should be hundreds of patients initiated on therapy, since January!
Otherwise. The company is unethical, immoral and corrupt. IMHO.
Approved in January... Tick, tock. Wouldn't you think PSTI would have been ready to go from the minute of approval, with 100's if not thousands, of patients that didn't qualify for studies?????
Think about it... Was the approval a big surprise?!?! CAREFUL, if it was a surprise, Leadership is incompetent.